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Advancing Childhood Cancer Research: The Impact and Significance of the RACE Act

 Last Update: 2023-01-06
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Although there are many targeted and immuno-oncology therapies for adult cancers, the development of new drugs for the unmet need for childhood cancer has been slow
.
For example, of the 36 drugs (including biosimilars) approved by the FDA for oncology indications in adults in 2018, 64% involved completed or ongoing trials
in the child population.
However, the median lag from the first human trial to the first pediatric trial of the final FDA-approved oncology drug was 6.
5 years, and only 5% of approved drugs included children
in initial approval.

There are two reasons for the delay in the development of childhood cancer drugs: first, drugs studied in children are largely selected based on adult indications, resulting in development patterns based on indications similar to those in adults, rather than on conditions that occur in both adults and children; Second, it takes a long time to start clinical trials in children, and the initiation also often depends on regulatory approval
for the adult indication.

There are two reasons for the lag in drug development in childhood cancer,

This article will excerpt the IQVIA Institute report, Advancing Childhood Cancer Research: Impact and Implications
.

1 Objectives of the RACE Act

The U.
S.
Accelerated Child Treatment and Equality Act (RACE Act) was passed by the U.
S.
Congress in 2017 and enacted
on August 18, 2020.
The legislation aims to accelerate early pediatric evaluation of adult cancer therapies under study and "ultimately facilitate the development of appropriate new therapies for pediatric patients.
"
This legislation requires pediatric research
of certain adult cancer-targeted drugs with new active ingredients based on molecular mechanisms of action rather than clinical indications.
This applies to treatments
targeting molecular targets identified by the FDA as "substantially associated with the onset or progression of pediatric cancer.
" The Act responds to
evidence that genetic and other molecular biological vulnerabilities of some adult cancers also exist in childhood cancers, and that up to half of childhood cancers may involve molecular abnormalities that can be treated with targeted drugs that have been approved for use in adults.

"substantially associated with the occurrence or progression of pediatric cancer"

2 Impact of the RACE Act

An analysis by the IQVIA Institute noted that nearly half of advanced cancer drug clinical trials have molecular targets defined by RACE
.

Nearly half

In addition, more than 70% of ongoing cancer trials involve drugs
with molecular targets defined by RACE.

70%

In trials using drugs with molecular targets defined by RACE, only 6.
9% included participants in the pediatric age group
.

6.
9%

Finally, IQVIA The Institute's analysis found that about 85 percent of trials sponsored by large (top 20) pharmaceutical companies were for drugs with molecular targets defined by RACE, compared with 65 percent
of trials sponsored by smaller companies.

85%65%

3 Challenges in developing cancer drugs in children that meet RACE requirements

Meeting RACE requirements due to the small number of childhood cancer patients, vulnerability, complexity of molecular target lists and exemption requirements, and the possibility of delayed and non-completion of pediatric drug research, such as the rarity of childhood cancers; pediatric patients have specific vulnerabilities and developmental problems; It is important to understand the list of pediatric molecular targets and exemptions and the possibility of delayed or non-completion of
paediatric research.

4 All stakeholders can contribute to the RACE Act

All clinical trial stakeholders – including regulators, patients and parents, oncology drug developers, pharmaceutical companies and policymakers – will play a key role
in achieving the goals of the RACE Act.

The RACE Act promotes systematic further collaboration, promotion, sharing, and connectivity
in oncology and pediatric scientific research.
To date, the biggest impact of the RACE Act has been to change expectations about the paradigm of pediatric oncology clinical development, emphasizing the need for partnerships among all stakeholders, including patient and parent groups, pharmaceutical companies, the FDA, pediatric oncology investigators, and national and international collaborative groups
.
The RACE Act has been successful
in this regard.
Other factors driving the success of legislation include:

The RACE Act promotes systematic further collaboration, promotion, sharing, and connectivity
in oncology and pediatric scientific research.
Other factors for success include:

Expand the scientific evidence base to identify biologically relevant molecular targets in childhood cancer and determine the potential applicability of new targeted oncology drugs being developed for adults
.

Give children with cancer a fair chance to access precision oncology treatments, including new targeted drugs, small molecules, immuno-oncology drugs, monoclonal antibodies, antibody-drug conjugates, bispecific antibodies, and cell and gene therapies
.

Reduce the time from the
first human trial to the first pediatric trial.

5 Next steps and priorities

As an FDA initiative, the RACE Act represents an important step forward for pediatric oncology, and the Global Cooperative Framework will drive its success
.
This legislation could bring benefits to clinical trials, particularly master protocols such as platform and basket trials; Overcoming barriers to data sharing among stakeholders; Advance the prioritization and sub-prioritization of molecular targets for pediatric oncology drugs
.

(1) Coordinate the consistency of international regulation

The FDA will play an important role in prioritizing drug development for children, potentially authorizing trials to study multiple therapies
at the same time, based on the expected level of approval.
From an industry perspective, cooperation between international regulators would be beneficial
.
This could help change the current status quo
where regulators give different answers to the same question.
The situation is reportedly improving, but full harmonization in the regulatory sense is unlikely unless there is direct access to legislators and policymakers around the
world.

(2) All stakeholders "work together"

Alignment is important
for better clinical trials to be consulted with all stakeholders.
By including the perspectives of all stakeholders, clinicians can be presented with the best possible trial protocol
.
This also provides the greatest opportunity to design trials that can be successfully and efficiently completed, and will provide sufficient data to characterize the drug and design meaningful endpoints, thus avoiding the need to repeat trials
in the future due to insufficient data.
Even if the outcome trial does not meet a certain remission rate or predetermined endpoint, the trial results can provide information and can help answer why the study did not meet the endpoint; What should be done differently next time; and what this result might tell about
other drugs in the class or other tumors of the same type and same diagnostic class.

(3) Enhance the role of patients and organizations, academia and CRO

The patient's and parents' perspectives are critical to future development, including what is most pressing and what level of risk is acceptable
.
There is also an opportunity for academia to play a greater role in advancing clinical trials, including defining and prioritizing drugs
that meet the greatest unmet patient needs.
This could gradually replace the current practice
of individual companies finding patient populations for specific drug trials.
In addition, CROs can help bridge the gap between clinical trial network and partner efforts, company needs, and the provision of regulatory-grade data to support new drug registration requirements; Other stakeholders may not have sufficient resources to conduct the necessary site monitoring and data quality checks
.
CROs can also help coordinate collaborative decision-making about which trials to conduct in specific rare patient populations, avoid duplication of effort, optimize efficiency, and can reduce the likelihood of
adult data being compromised.

Patient and parent academic CRO

(4) Main goal: to help children with cancer

All stakeholders share a common goal of improving existing treatments
for children with cancer.
With early engagement and collaboration, the expertise of each stakeholder group can advance pediatric oncology
as efficiently and effectively as possible.
In addition to incentivizing these underserved patient populations, regulatory incentives such as exclusivity or other potential measures to stimulate drug development can also be implemented after early initial evaluation of drug
candidates.
Once a drug enters a phase I trial, early discussions
with experts should begin as soon as possible.
This will give the company enough time to generate interest on the drug and then discuss with experts whether to apply for an exemption
.
The legislative purpose of the RACE Act is not to increase the number of trials, but to more carefully target trials based on earlier
evaluations.

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